An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia

AstraZeneca315 enrolled

Overview

This study is being carried out to see if the study medication, rosuvastatin, is effective in treating familial hypercholesterolaemia in children and adolescents, and to determine the long term (over 2 years) safety, tolerability and efficacy of the study medication in these patients. This study will also measure levels of drug in the blood and see how well it is tolerated. This is known as pharmacokinetic (PK) analysis. At baseline only a small number of patients will participate in a single dose PK phase over 24 hours. In order to see if this medication works, a control group of healthy siblings will help the researchers to compare certain results.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

315

Conditions

Familial Hypercholesterolaemia

Interventions

Eligibility

Sex: ALLMin age: 6 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * children and adolescents (aged 6 to less than 18 years) with Familial Hypercholesterolaemia * Patients aged between 6 and less than 10 years of age must not be taking a statin medicine Exclusion Criteria: * History of muscle or sensitivity reactions to any statin medicines * Current active liver disease or dysfunction (except a confirmed diagnosis of Gilbert's disease)

Locations (15)

Research Site

Cincinnati, Ohio, United States

Research Site

Leuven, Belgium, Belgium

Research Site

Leuven, Belgium

Research Site

Vancouver, British Columbia, Canada

Research Site

Hamilton, Ontario, Canada

Research Site

Toronto, Ontario, Canada

Research Site

Chicoutimi, Quebec, Canada

Research Site

Québec, Quebec, Canada

Research Site

Amsterdam, Netherlands

Research Site

Groningen, Netherlands

...and 5 more locations

Outcomes

Primary Outcomes

Percent Change From Baseline in LDL-C

Negative values represent a decrease and positive values represent an increase. In total, 198 patients were treated. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Time frame: At Month 3, Month 12 and Month 24

Sexual Maturation by Tanner Staging at Baseline

Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.

Time frame: At Baseline

Single Dose PK - Cmax

Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing

Time frame: Serial blood samples over 24 hours.

Percent Change From Baseline in Height

One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Time frame: At Month 12 and Month 24

Sexual Maturation by Tanner Staging at Month 12

Time frame: At Baseline

Sexual Maturation by Tanner Staging at Month 24

Time frame: At Baseline

Single Dose PK - Tmax

Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing

Time frame: Serial blood samples over 24 hours

Single Dose PK - AUC(0-24)

Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing

Time frame: Serial blood samples over 24 hours

Secondary Outcomes

Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1

One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Time frame: At Month 3, Month 12 and Month 24

Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)

One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Time frame: At Month 12 and Month 24

Adverse Events

Number of participants with Various Categories of AE's. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Time frame: 2-year study period

Total Duration of Exposure

Total duration of exposure was calculated as \[last dose date of rosuva - first dose date of rosuva + 1 day\]. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Time frame: 2-year study period

Overal Treatment Adherence

Overall adherence rate was calculated as the weighted mean of adherence rates of all consecutive visits after baseline, in which the adherence rate between 2 consecutive visits was a percentage of the number of rosuvastatin taken divided by duration of exposure. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.

Time frame: 2-year study period